Leverage multiple real-world data sources in single-arm medical device clinical studies.
J Biopharm Stat
; 32(1): 107-123, 2022 01 02.
Article
em En
| MEDLINE
| ID: mdl-33844621
ABSTRACT
The interest in utilizing real-world data (RWD) has been considerably increasing in medical product development and evaluation. With proper usage and analysis of high-quality real-world data, real-world evidence (RWE) can be generated to inform regulatory and healthcare decision-making. This paper proposes a study design and data analysis approach for a prospective, single-arm clinical study that is supplemented with patients from multiple real-world data sources containing patient-level covariate and outcome data. After the amount of information to be borrowed from each real-world data source is determined, the propensity score-integrated composite likelihood method is applied to obtain an estimate of the parameter of interest based on data from the prospective clinical study and this real-world data source. This method is applied to each real-world data source. The final estimate of the parameter of interest is then obtained by taking a weighted average of all these estimates. The performance of the proposed approach is evaluated via a simulation study. A hypothetical example is presented to illustrate how to implement the proposed approach.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Projetos de Pesquisa
/
Armazenamento e Recuperação da Informação
Tipo de estudo:
Observational_studies
/
Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
J Biopharm Stat
Assunto da revista:
FARMACOLOGIA
Ano de publicação:
2022
Tipo de documento:
Article
País de afiliação:
Estados Unidos